The decline of pharma R&D productivity (2)

Science needs to move beyond luck if it is to design better drugs for the brain

The Economist:

Between 2011 and 2020 the likelihood of a drug in psychiatry being approved by the Food and Drug Administration was 7.3%. In neurology it was 5.9%. (The industry average is 7.9%.) As well as being less likely to succeed in trials (see chart), neurology drugs also take much longer, on average, to develop, further decreasing their appeal

According to the Global Burden of Disease project 12 mental-health disorders affect about 970m people. Their prevalence has increased by 48% since 1990 as the population has grown. With more than one in ten people on the planet affected, it is a global problem, although what data are available suggest it is more marked in Western countries (see map).

Paying for rare diseases drugs

The next generation of rare disease drug policy: ensuring both innovation and affordability

The study finds that while 5% of drugs with an orphan indication cost more than $500,000 per year in US, these drugs make up only 0.08% of all patients treated by drugs with an orphan indication. The majority (52%) of patients who receive treatment an orphan drug get the treatment for <$50,000.

Pharmaceutical contracts and prices

  Introduction to Market Access for Pharmaceuticals

Contents:

Chapter 1: Health as a Good

Chapter 2: Decision-Making in Public Health

Chapter 3: Definition and Concepts

Chapter 4: HTA Decision Analysis framework

Chapter 5: Early HTA Advice

Chapter 6: Overview of Market Access Agreements

Chapter 7: External Reference Pricing

Chapter 8: Gap between Payers and Regulators

Chapter 9: Early Access Programs

Chapter 10: Market Access of Orphan Drugs

Building a think tank

 Build a think tank. A guide for policy entrepreneurs

Using this guide 8
The reasons for creating this companion guide 8
Using this companion guide 8
Introduction 11
The importance of supporting new think tank development 11
Unravelling the definition of think tanks 13
What’s in the name? 13
Defining think tanks 14
Think tank functions 15
Summing up 16
The Why? questions 18
Why do you want to set up a think tank? 18
Why do think tanks aim to influence policy? 23
Are you sure you want to establish a think tank? 25
The What? questions 26
What will your think tank do? 26
What is the context? 27
What do you want to achieve by setting up a think tank? 33
What issues will the think tank focus on? 37
What will the think tank want to influence? 38
What will its business model be? 40
The Who? questions 46
Who will govern it? (And how?) 46
Who will lead the think tank? 51
Who will engage with it? 56
Who will work for it? 60
Who will fund it? 65
Who will support it? 71
The How? questions 72
How will it carry out research? 72
How will it be managed? 79
How will it communicate? 82
How will it monitor its progress? 88
How will you ensure its credibility? 92
How will it adapt to change? 95
How will it engage with evolving technology? 99
The ‘When’? questions 101
When to start? 101
When to let go? 106
Checklist: Establishing a think tank 108
References and resources 110
On the definition and functions of think tanks 110
Boards and governance 111
Creating and managing think tanks 111
Policy impact 111
Communications 112
Funding and financial management 112
Context 113
Evidence-informed policy 113
List of Boxes, Figures and Tables 114

https://buildathinktank.org/

Market access for expensive therapies (or how to overcome prices) (2)

 Gene and Cell Therapies-Market Access and Funding

Contents:

Chapter 1        Introduction to cell and gene therapies concepts and definitions in US and EU

Chapter 2        cell and gene therapies: genuine products and potential for dramatic value

Chapter 3        cell and gene therapies: Regulatory aspects in US and EU

Chapter 4        the need for new HTA reference case for cell and gene therapies

Chapter 5        How to mitigate cell and gene therapies uncertainties and HTA risk adverse attitude?

Chapter 6        Cell and gene therapies funding: challenges and solutions for patients’ access

Chapter 7        Conclusion

Market access for expensive therapies (or how to overcome prices)

 Managed Entry Agreements and Funding for Expensive Therapies

Key reference to understand what's going on in this topic. Recommended.

Contents:

1. Introduction to Managed Entry Agreements

2. Definition and Classification of MEAs

3. From Coverage with Evidence Development to Individual Performance-Based Agreements in Italy

4. Coverage with Evidence Development for Multiple Sclerosis Drugs in the UK: A "Costly Failure"?

5. Country Comparison of the Implementation of Managed Entry Agreements

6. Novel Funding Models for Expensive Therapies

7. Managed Entry Agreement for Cell and Gene Therapies

The decline of pharma R&D productivity (how much are we willing to pay for failure (95%) or success (5%)?)

Global Trends in R&D . Overview through 2021 

The productivity of the clinical development process can be considered as a measure of trial outputs (drugs, innovation, trial success, etc.), compared to a measure of trial inputs or resources dedicated to obtaining those outputs (e.g., aspects of trial complexity, duration, monetary investments, etc.). Such measures of success, complexity and trial duration were selected for inclusion in the productivity index as described above. Increases in success will increase productivity overall as will decreases in complexity or duration. Conversely, decreases in success will drive down the productivity index, as do increases in complexity and duration.

Clinical development productivity — a composite metric of success rates, clinical trial complexity and trial duration — declined in 2021, continuing an overarching 10-year trend. Despite a second year of decreasing trial complexity, the ongoing decline in success rates has resulted in reduced average trial productivity. Trial success rates fell to their lowest in more than 10 years to an average 5% likelihood of progressing successfully through all phases.

Health systems design

 Building a High- Value Health System

Countries and institutions worldwide face the challenge of planning and paying for health care that effectively meets the needs of citizens and employees. While there are many criticisms of existing healthcare models, current literature offers little guidance for individuals who want to carry out the work of redesigning and improving their health system.

Pharma, big pharma (15)

Sick Money. The Truth About the Global Pharmaceutical Industry 

Pharmaceutical innovation and value extraction

 Pharmaceutical innovation sourcing

Figure below shows that 23% of new medicines came from public bodies and private-private collaboration and they didn't apply for any marketing authorisation.

Managing the decline in Pharma R&D

Global Trends in R&D. OVERVIEW THROUGH 2021

A decade ago I posted this: Gestionar el declivi. John Kay said in 2011:

"When an industry model is broken, the best business strategy may be to manage its decline"

Now a new report confirms that the process continues after a decade.

From IQVIA report:

The composite success rate across all development phases and therapy areas declined to 5.0% in 2021, which can be attributed to an appetite for increased scientific risk in clinical development programs as the bar for efficacy and safety rises, as well as increased pauses in product development due to the pandemic.

Across disease areas, probability of success varies considerably, and 2021’s composite success rate fell below the 10-year trend in all areas except for vaccines and cardiovascular.

 That's all folks.

Human genomics vs. clinical genomics

 Today my suggestion is to read the post by Eric Topol with the same title. 

It begins with this statement:

We’re now well over 20 years since the first human genome was sequenced, but with few exceptions the massive amount of data that has been generated has not been transformed to routine patient care.

So, why?

Setting priorities explicitly (or not) (2)

 REASONABLE RATIONING. International Experience of Priority Setting in Health Care

COVID and patents

Unprecedented. The Rapid Innovation Response to COVID-19 and the Role of Intellectual Property